, Volume 36, Issue 7, pp 733–743 | Cite as

Personalized Medicine and Pay for Performance: Should Pharmaceutical Firms be Fully Penalized when Treatment Fails?

  • Fernando AntoñanzasEmail author
  • Roberto Rodríguez-Ibeas
  • Carmelo A. Juárez-Castelló
Leading Article


In this article, we model the behavior of a pharmaceutical firm that has marketing authorization for a new therapy believed to be a candidate for personalized use in a subset of patients, but that lacks information as to why a response is seen only in some patients. We characterize the optimal outcome-based reimbursement policy a health authority should follow to encourage the pharmaceutical firm to undertake research and development activities to generate the information needed to effectively stratify patients. Consistent with the literature, we find that for a pharmaceutical firm that does not undertake research and development activities, when the treatment fails, the total price of the drug must be returned to the healthcare system (full penalization). By contrast, if the firm undertakes research and development activities that make the implementation of personalized medicine possible, treatment failure should not be fully penalized. Surprisingly, in some cases, particularly for high-efficacy drugs and small target populations, the optimal policy may not require any penalty for treatment failure. To illustrate the main results of the analysis, we provide a numerical simulation and a graphical analysis.

JEL Classification

I11 I18 



Financial support from MINECO (Project ECO2016-78685-R) is gratefully acknowledged. We thank Paul Overton from Beacon Medical Communications (UK) for the English editing of this manuscript. We thank the editor and two anonymous referees for their comments and suggestions.

Author Contributions

RR-I acted as a health economist on this article, developed the model analytical results, and contributed to the writing of the text. CAJ-C acted as a health economist on this article, programmed and ran the numerical simulations, and designed the figures. FA acted as a health economist on this article, conceptualized the research problem, contributed to the writing of the test, and acted as the overall guarantor for the overall content of this article. All authors contributed to the conception and planning of the work, and critically revised and approved the final submitted version of the manuscript.

Compliance with Ethical Standards


This study was funded by the Spanish Ministry of Economics, MINECO (Project ECO2016-78685-R).

Conflict of interest

Fernando Antoñanzas, Carmelo A. Juárez-Castelló, and Roberto Rodríguez-Ibeas have no conflicts of interest directly relevant to the content of this article.

Supplementary material

40273_2018_619_MOESM1_ESM.xlsx (265 kb)
Supplementary material 1 (XLSX 264 kb)


  1. 1.
    Redekop WK, Mladsi D. The faces of personalized medicine: a framework for understanding its meaning and scope. Value Health. 2013;16:54–9.CrossRefGoogle Scholar
  2. 2.
    Antoñanzas F, Juárez-Castelló C, Rodríguez-Ibeas R. Some economics on personalized and predictive medicine. Eur J Health Econ. 2015;16(9):985–94.CrossRefPubMedGoogle Scholar
  3. 3.
    Annemans L, Redekop K, Payne K. Current methodological issues in the economic assessment of personalized medicine. Value Health. 2013;16(6 Suppl.):S20–6.CrossRefPubMedGoogle Scholar
  4. 4.
    Sairamesh J, Rossbach M. An economic perspective on personalized medicine. Hugo J. 2013;7(1):1–2.CrossRefGoogle Scholar
  5. 5.
    Connor S. Our Drugs Do Not Work On Most Patients. The Independent (London), 13 Dec 2011. Accessed 6 Feb 2018.
  6. 6.
    Culbertson AW, Valentine SJ, Naylor S. Personalized medicine: technological innovation and patient empowerment or exuberant hyperbole? Drug Discov World. 2007;8(3):18–32.Google Scholar
  7. 7.
    Trusheim MR, Berndt ER, Douglas FL. Stratified medicine: strategic and economic implications of combining drugs and clinical biomarkers. Nat Rev Drug Discov. 2007;6(4):287–93.CrossRefPubMedGoogle Scholar
  8. 8.
    Jain S, Shankaran V. The economics of personalized therapy in metastatic colorectal cancer. Curr Colorectal Cancer Rep. 2016;12:123–9.CrossRefGoogle Scholar
  9. 9.
    Lièvre A, Bachet JB, Le Corre D, Boige V, Landi B, Emile JF, Côté JF, Tomasic G, Penna C, Ducreux M, Rougier P, Penault-Llorca F, Laurent-Puig P. KRAS mutation status is predictive of response to cetuximab therapy in colorectal cancer. Cancer Res. 2006;66(8):3992–5.CrossRefPubMedGoogle Scholar
  10. 10.
    Behl AS, Goddard KA, Flottemesch TJ, Veenstra D, Meenan RT, Lin JS, Maciosek MV. Cost-effectiveness analysis of screening for KRAS and BRAF mutations in metastatic colorectal cancer. J Natl Cancer Inst. 2012;104(23):1785–95.CrossRefPubMedPubMedCentralGoogle Scholar
  11. 11.
    Thierry AR, Mouliere F, El Messaoudi S, Mollevi C, Lopez-Crapez E, Rolet F, Gillet B, Gongora C, Dechelotte P, Robert B, Del Rio M, Lamy PJ, Bibeau F, Nouaille M, Loriot V, Jarrousse AS, Molina F, Mathonnet M, Pezet D, Ychou M. Clinical validation of the detection of KRAS and BRAF mutations from circulating tumor DNA. Nat Med. 2014;20:430–5.CrossRefPubMedGoogle Scholar
  12. 12.
    Carlson JJ, Garrison LP, Ramsey SD, Veenstra DL. The potential clinical and economic outcomes of pharmacogenomic approaches to EGFR-tyrosine kinase inhibitor therapy in non-small-cell lung cancer. Value Health. 2009;12(1):20–7.CrossRefPubMedGoogle Scholar
  13. 13.
    NICE. Erlotinib and gefitinib for treating non-small-cell lung cancer that has progressed after prior chemotherapy. 2015. Accessed 9 Jan 2018.Google Scholar
  14. 14.
    Faulkner E, Annemans L, Garrison L, Helfand M, Holtorf AP, Hornberger J, Hughes D, Li T, Malone D, Payne K, Siebert U, Towse A, Veenstra D, Watkins J, Personalized Medicine Development and Reimbursement Working Group. Challenges in the development and reimbursement of personalized medicine: payer and manufacturer perspectives and implications for health economics and outcomes research: a report of the ISPOR Personalized Medicine Special Interest Group. Value Health. 2012;15(8):1162–71.CrossRefPubMedGoogle Scholar
  15. 15.
    Carlson JJ, Chen S, Garrison LP. Performance-based risk-sharing arrangements: an updated international review. Pharmacoeconomics. 2017;35:1063–72.CrossRefPubMedGoogle Scholar
  16. 16.
    Towse A, Garrison LP. Can’t get no satisfaction? Will pay for performance health? Pharmacoeconomics. 2010;28(2):93–102.CrossRefPubMedGoogle Scholar
  17. 17.
    de Pouvourville G. Risk-sharing agreements for innovative drugs. Eur J Health Econ. 2006;7:155–7.CrossRefPubMedGoogle Scholar
  18. 18.
    Pita-Barros P. The simple economics of risk-sharing agreements between the NHS and the pharmaceutical industry. Health Econ. 2011;20:461–70.CrossRefGoogle Scholar
  19. 19.
    Zaric GS, O’Brien BJ. Analysis of a pharmaceutical risk sharing agreement based on the purchaser’s total budget. Health Econ. 2005;14:793–803.CrossRefPubMedGoogle Scholar
  20. 20.
    Zaric GS, Xie B. The impact of two pharmaceutical risk-sharing agreements on pricing, promotion and net health benefits. Value Health. 2009;12(5):838–45.CrossRefPubMedGoogle Scholar
  21. 21.
    Antoñanzas F, Juaréz-Castelló C, Rodríguez-Ibeas R. Should health authorities offer risk-sharing contracts to pharmaceutical firms? A theoretical approach. Health Econ Policy Law. 2011;6:391–403.CrossRefPubMedGoogle Scholar
  22. 22.
    Towse A, Garrison LP. Economic incentives for evidence generation: promoting an efficient path to personalized medicine. Value Health. 2013;16(6 Suppl.):S39–43.CrossRefPubMedGoogle Scholar
  23. 23.
    O’Donnell JC. Personalized medicine and the role of health economics and outcomes research: issues, applications, emerging trends, and future research. Value Health. 2013;16(6 Suppl.):S1–3.CrossRefPubMedGoogle Scholar
  24. 24.
    Pita-Barros P, Martínez-Giralt X. Health economics: an industrial organization perspective. London, New York: Routledge, Taylor and Francis Group; 2012.Google Scholar
  25. 25.
    Trusheim MR, Burgess B, Hu SX, Long T, Averbuch SD, Flynn AA, et al. Quantifying factors for the success of stratified medicine. Nat Rev Drug Discov. 2011;10(11):817–33.CrossRefPubMedGoogle Scholar
  26. 26.
    Satanove D. The challenging economics of the companion diagnostic industry; a compelling case for patent protection. J Intell Prop Ent Law. 2016;6(1):142–71.Google Scholar
  27. 27.
    Graf von der Schulenburg JM. Frank M. Rare is frequent and frequent is costly: rare diseases as a challenge for health care systems. Eur J Health Econ. 2015;16(2):113–8.CrossRefPubMedGoogle Scholar

Copyright information

© Springer International Publishing AG, part of Springer Nature 2018

Authors and Affiliations

  1. 1.Department of EconomicsUniversity of La RiojaLogroñoSpain

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